Anthony S. Stein
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View article: Allogeneic hematopoietic stem cell transplantation for acute leukemias of mixed or ambiguous lineage
Allogeneic hematopoietic stem cell transplantation for acute leukemias of mixed or ambiguous lineage Open
Acute leukemias of ambiguous lineage (ALAL) are characterized by ≥ 20% abnormal progenitors without clear differentiation along a single lineage or with commitment to more than 1 lineage. ALAL includes acute undifferentiated leukemia (AUL)…
View article: A multicenter phase 1 trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after allogeneic hematopoietic cell transplant in patients with KMT2A-rearranged or NPM1-mutated acute leukemia
A multicenter phase 1 trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance after allogeneic hematopoietic cell transplant in patients with KMT2A-rearranged or NPM1-mutated acute leukemia Open
Background and Significance Allogeneic stem cell transplant (alloSCT) is a potentially curative post-remission therapy for patients with acute leukemia; however, chromosomal rearrangements involving the lysine methyltransferase 2A (KMT2A) …
View article: Favorable outcomes including post-transplant survival are seen independent of baseline skin burden in treatment-naïve patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG): Subgroup analysis of a pivotal Trial
Favorable outcomes including post-transplant survival are seen independent of baseline skin burden in treatment-naïve patients (pts) with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG): Subgroup analysis of a pivotal Trial Open
Introduction BPDCN is an aggressive orphan malignancy characterized by CD123 expression and involvement of the skin, bone marrow (BM), blood, and visceral organs. TAG, a first-in-class CD123-targeted therapy, is the only agent approved for…
View article: Longer survival with tagraxofusp versus venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): Results of a real-world analysis
Longer survival with tagraxofusp versus venetoclax in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN): Results of a real-world analysis Open
Background BPDCN is an orphan and aggressive hematologic malignancy characterized by CD123 expression and frequent involvement of the skin, bone marrow, blood, lymph nodes, liver, and spleen. An objective of first-line (1L) therapy is to i…
View article: Day 7 mount sinai acute GVHD international consortium (MAGIC) algorithm probability predicts GVHD and non-GVHD-related non-relapse mortality in a contemporary cohort of allogeneic transplant patients
Day 7 mount sinai acute GVHD international consortium (MAGIC) algorithm probability predicts GVHD and non-GVHD-related non-relapse mortality in a contemporary cohort of allogeneic transplant patients Open
The Mount Sinai Acute GVHD International Consortium (MAGIC) developed and validated the MAGIC algorithm probability (MAP), which is a biomarker-based risk model using serum levels of ST2 and REG3α as predictive biomarkers of acute graft-ve…
View article: Phase I first-in-human dose escalation study of the oral casein kinase 1α and cyclin dependent kinase 7/9 inhibitor BTX A51 in advanced MDS and AML
Phase I first-in-human dose escalation study of the oral casein kinase 1α and cyclin dependent kinase 7/9 inhibitor BTX A51 in advanced MDS and AML Open
NCT04872166.
View article: Genetic characteristics of blastic plasmacytoid dendritic cell neoplasm: A single institution experience
Genetic characteristics of blastic plasmacytoid dendritic cell neoplasm: A single institution experience Open
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with poorly characterized molecular features. To identify disease-specific mutational profiles, we performed targeted next-generation sequencing (NGS) …
View article: Targeted Irradiation and STAT3 Inhibition Reprogram the AML Microenvironment and Extend Survival: Toward Translational Immunoradiotherapy
Targeted Irradiation and STAT3 Inhibition Reprogram the AML Microenvironment and Extend Survival: Toward Translational Immunoradiotherapy Open
Purpose Despite advances in acute myeloid leukemia (AML) therapy, relapses remain challenging. While AML is radiation-sensitive, total body irradiation (TBI) causes organ toxicities and activates tolerogenic/proangiogenic STAT3 signaling. …
View article: Pulmonary manifestations during neutrophil engraftment in recipients of allogeneic hematopoietic cell transplantation for myelofibrosis
Pulmonary manifestations during neutrophil engraftment in recipients of allogeneic hematopoietic cell transplantation for myelofibrosis Open
Allogeneic hematopoietic cell transplantation (alloHCT) remains the only potentially curative treatment for both primary and secondary myelofibrosis (MF). However, outcomes are often complicated by the presence of cardiopulmonary comorbidi…
View article: Treatment of Relapsed/Refractory <scp>AML</scp>—Novel Treatment Options Including Immunotherapy
Treatment of Relapsed/Refractory <span>AML</span>—Novel Treatment Options Including Immunotherapy Open
Acute myeloid leukemia is a molecularly heterogenous disease caused by the rapid expansion and impaired differentiation of malignant myeloid progenitors. Overall, outcomes remain poor, and more than half of patients develop relapsed or ref…
View article: <i>TP53</i> mutations are associated with CD19− relapse and inferior outcomes after blinatumomab in adults with ALL
<i>TP53</i> mutations are associated with CD19− relapse and inferior outcomes after blinatumomab in adults with ALL Open
Despite the success of the CD19 × CD3 T-cell engager blinatumomab in B-cell acute lymphoblastic leukemia (B-ALL), treatment failure is common and can manifest with antigen loss and extramedullary disease (EMD) relapse. To understand the im…
View article: Combined Cytokine Blockade Therapy (CCBT) Using Basiliximab and Infliximab for Treatment of Steroid-Refractory Graft-Versus-Host Disease (SR-GvHD)
Combined Cytokine Blockade Therapy (CCBT) Using Basiliximab and Infliximab for Treatment of Steroid-Refractory Graft-Versus-Host Disease (SR-GvHD) Open
Background: The standard first-line treatment for acute graft-versus-host disease (aGvHD) is systemic, high-dose glucocorticoids which have historically had limited responses. Combined cytokine blockade therapy (CCBT) with the monoclonal a…
View article: Venetoclax in combination with a pediatric-inspired regimen for the treatment of newly diagnosed adults with Philadelphia chromosome-negative acute lymphoblastic leukemia
Venetoclax in combination with a pediatric-inspired regimen for the treatment of newly diagnosed adults with Philadelphia chromosome-negative acute lymphoblastic leukemia Open
BCL-2 protein overexpression, common in B-cell acute lymphoblastic leukemia (B-ALL), including the Philadelphia (Ph)-like subtype, mediates leukemic cell survival. We treated 24 patients with 14 days of BCL-2 inhibitor, venetoclax, 400 mg …
View article: Phase 1/2, Open-Label, Multi-Center Study Assessing the Safety, Tolerability and Preliminary Efficacy of CD123 Natural Killer Cell Engager (NKCE), SAR443579, in Combination with Venetoclax and Azacitidine in Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy
Phase 1/2, Open-Label, Multi-Center Study Assessing the Safety, Tolerability and Preliminary Efficacy of CD123 Natural Killer Cell Engager (NKCE), SAR443579, in Combination with Venetoclax and Azacitidine in Patients with Newly Diagnosed Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy Open
Background and Significance: SAR443579 (SAR'579) is an NKCE composed of three distinct binding domains designed to co-engage CD123 on tumor cells and the activating receptors NKp46 and CD16a on natural killer (NK) cells. CD123 is highly ex…
View article: Safety of 20Gy Total Marrow Irradiation Based Conditioning Regimen for Hematopoietic Cell Transplantation with T Cell Adoptive Immunotherapy
Safety of 20Gy Total Marrow Irradiation Based Conditioning Regimen for Hematopoietic Cell Transplantation with T Cell Adoptive Immunotherapy Open
Introduction Acute leukemia patients with chemorefractory active disease at the time of hematopoietic stem cell transplantation (HSCT) have high-risk of post-transplant relapse and a poor prognosis. Indeed, utility of HSCT has been questio…
View article: TP53 Mutations Increased the Risk of CD19 Negative Relapse Following Blinatumomab in Adults with B-Cell Acute Lymphoblastic Leukemia (B-ALL)
TP53 Mutations Increased the Risk of CD19 Negative Relapse Following Blinatumomab in Adults with B-Cell Acute Lymphoblastic Leukemia (B-ALL) Open
Introduction: Blinatumomab (blina) is a bispecific CD3xCD19 T cell engager that redirects autologous T cells to target CD19+ lymphoblasts. Despite the success of blina in B-ALL, treatment failure is common and can manifest with unique patt…
View article: Tissue-Specific Myeloablative Conditioning with Total Marrow Irradiation Enhances Gut Microbiome Diversity and Mitigates Gvhd: A Novel Therapeutic Approach
Tissue-Specific Myeloablative Conditioning with Total Marrow Irradiation Enhances Gut Microbiome Diversity and Mitigates Gvhd: A Novel Therapeutic Approach Open
Background: Traditional systemic myeloablative conditioning methods, such as total body irradiation (TBI) and chemotherapy, severely damage the gastrointestinal (GI) tract and induce graft-versus-host disease (GVHD), negatively impacting p…
View article: Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study
Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study Open
Introduction Treatment of myelodysplastic syndrome (MDS) with primary refractory disease or relapse after frontline treatment (r/r MDS) represents a high unmet medical need. Hypomethylating (HMA) agents, including azacitidine, are used in …
View article: Ivosidenib in Patients with Mutant Isocitrate Dehydrogenase 1 (m<i>IDH1</i>) Relapsed/Refractory Myelodysplastic Syndromes: Update of the Phase 1 Substudy per the International Working Group 2023 Response Criteria
Ivosidenib in Patients with Mutant Isocitrate Dehydrogenase 1 (m<i>IDH1</i>) Relapsed/Refractory Myelodysplastic Syndromes: Update of the Phase 1 Substudy per the International Working Group 2023 Response Criteria Open
Introduction Patients (pts) with relapsed/refractory (R/R) myelodysplastic syndromes (MDS) have a poor prognosis and, until recently, lacked therapeutic options. Ivosidenib (IVO) is an oral, targeted, small-molecule inhibitor of mIDH1. In …
View article: Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in <i>NPM1</i>-mutant AML
Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in <i>NPM1</i>-mutant AML Open
Although intensive induction chemotherapy (IC) remains the standard of care for younger patients with acute myeloid leukemia (AML), hypomethylating agents + venetoclax (HMA/VEN) can lead to durable remission among older patients with nucle…
View article: Tyrosine kinase inhibitor maintenance following chimeric antigen receptor T‐cell therapy in Philadelphia chromosome‐positive acute lymphoblastic leukaemia
Tyrosine kinase inhibitor maintenance following chimeric antigen receptor T‐cell therapy in Philadelphia chromosome‐positive acute lymphoblastic leukaemia Open
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View article: Outcomes following allogeneic hematopoietic cell transplantation relapse in Philadelphia chromosome‐positive acute lymphoblastic leukemia
Outcomes following allogeneic hematopoietic cell transplantation relapse in Philadelphia chromosome‐positive acute lymphoblastic leukemia Open
Improved first progression-free survival following allogeneic hematopoietic cell transplantation relapse with the use of immunotherapy.
View article: BRAF Mutations in Patients with Myeloid Neoplasms: A Cancer Center Multigene Next-Generation Sequencing Analysis Experience
BRAF Mutations in Patients with Myeloid Neoplasms: A Cancer Center Multigene Next-Generation Sequencing Analysis Experience Open
BRAF mutations are rare in myeloid neoplasms and are reported to be associated with poor treatment outcomes. The purpose of our study is to characterize BRAF mutations in myeloid neoplasms using a next-generation sequencing (NGS) panel bas…
View article: Final phase 1 substudy results of ivosidenib for patients with mutant <i>IDH1</i> relapsed/refractory myelodysplastic syndrome
Final phase 1 substudy results of ivosidenib for patients with mutant <i>IDH1</i> relapsed/refractory myelodysplastic syndrome Open
Ivosidenib is a first-in-class mutant isocitrate dehydrogenase 1 (mIDH1) inhibitor with efficacy and tolerability in patients with advanced mIDH1 hematologic malignancies, leading to approval in frontline and relapsed/refractory (R/R) mIDH…